Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes

Article ID	Journal	Published Year	Pages	File Type
8366227	Steroids	2018	42 Pages	PDF

Abstract

Vamorolone is a dissociative steroid that retains high affinity binding and nuclear translocation of both glucocorticoid (agonist) and mineralocorticoid (antagonist) receptors, but does not show pharmacodynamic safety concerns of existing glucocorticoid drugs at up to 20â¯mg/kg/day.

Keywords

NOAEL NCATS ULN SAE CBG 11β-hydroxysteroid dehydrogenase SRC ALT MTD ACTH GRE MAD NIH AUC CL/F TEAE Alanine aminotransferase electrocardiogram ECG Upper limit of normal Maximum tolerated dose body mass index BMI adverse event Glucocorticoid response element severe adverse event Treatment-emergent adverse events SAD Pharmacokinetics National Institutes of Health Principal investigator ICH area under the curve adrenocorticotropic hormone cortisol binding globulin glucocorticoid receptor

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Life Sciences Biochemistry, Genetics and Molecular Biology Biochemistry

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Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes

Authors

Eric P. Hoffman, Valerie Riddle, Maxime A. Siegler, Daniel Dickerson, Miroslav Backonja, William G. Kramer, Kanneboyina Nagaraju, Heather Gordish-Dressman, Jesse M. Damsker, John M. McCall,